Lucerastat for untreated Fabry disease in men
Part of Brain & nervous system, Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.
This trial tests a new medicine called lucerastat for men with Fabry disease who have not yet received treatment. The goal is to see if it can slow down or improve symptoms and whether it is safe.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a confirmed diagnosis of Fabry disease, with very low enzyme activity or a specific gene change.
- You have had at least one symptom of Fabry disease, such as nerve pain, cornea verticillata (a cloudy pattern in the eye), or angiokeratomas (small red or purple spots on the skin).
- You have not had any treatment for Fabry disease in the last 6 months, or ever.
- Your blood test shows a certain level of a substance called lyso-Gb3 (at least 20 ng/ml).
- Your kidneys are working well enough (eGFR of 45 or higher).
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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